3. Spinal muscular atrophy Clinical trials / Disease details
Clinical trials : 217 / Drugs : 149 - (DrugBank : 33) / Drug target genes : 54 - Drug target pathways : 80
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02268552 (ClinicalTrials.gov) | April 2, 2015 | 1/10/2014 | An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) | An Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: branaplam | Novartis Pharmaceuticals | NULL | Active, not recruiting | N/A | 182 Days | All | 40 | Phase 1/Phase 2 | Belgium;Bulgaria;Denmark;Germany;Italy;Poland;Russian Federation;Czechia;Hungary;Netherlands |
| 2 | EUCTR2014-002053-19-BE (EUCTR) | 15/12/2014 | 04/11/2014 | Clinical trial of LMI070 given by mouth to Type I SMA infant patients. | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 INN or Proposed INN: Branaplam Other descriptive name: LMI070 | Novartis Pharma Services AG | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 1;Phase 2 | United States;Hungary;Czech Republic;Poland;Belgium;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy | ||
| 3 | EUCTR2014-002053-19-PL (EUCTR) | 03/01/2018 | Clinical trial of LMI070 given by mouth to Type I SMA infant patients. | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: LMI070 INN or Proposed INN: Branaplam Other descriptive name: LMI070 | Novartis Pharma Services AG | NULL | NA | Female: yes Male: yes | 44 | Phase 1;Phase 2 | United States;Hungary;Czech Republic;Belgium;Poland;Denmark;Bulgaria;Russian Federation;Netherlands;Germany;Italy |